Novartis's Fabhalta Phase III Success Prompts FDA Priority Review in IgAN

Swiss pharmaceutical giant Novartis has reported promising Phase III clinical trial results for its drug Fabhalta (iptacopan), which targets kidney disease. The drug has shown a significant reduction in proteinuria, a condition characterized by the presence of excess protein in urine, which is a key indicator of kidney disease. In October 2023, the study achieved its primary endpoint in an interim analysis. The latest data reveal that Fabhalta-treated patients experienced a 38.3% decrease in proteinuria after nine months, a substantial improvement over the placebo group.

The Phase III trial involved 518 participants who were given either Fabhalta or a placebo alongside standard supportive care. With these results, Novartis has submitted an application for accelerated approval from the FDA, which has been accepted for priority review. Although the FDA has not yet set a deadline for its decision, the standard six-month review timeline suggests a decision could be expected by the end of 2024.

It is noteworthy that while the drug has been linked to a reduction in proteinuria, its impact on kidney function has not been confirmed. The FDA has previously granted accelerated approval to other IgA nephropathy (IgAN) drugs based on proteinuria data alone. For instance, Travere Therapeutics' Filspari (sparsentan) was approved after demonstrating a 45% reduction in proteinuria, and Calliditas Therapeutics' Tarpeyo showed a 34% reduction.

Novartis is also conducting a long-term study to assess the impact of Fabhalta on kidney function over a period of 24 months. The company anticipates completing this study and obtaining data on its second primary endpoint in 2025. The potential accelerated approval for Fabhalta in IgAN is part of Novartis' strategy to achieve substantial sales for the drug. In December 2023, the FDA approved Fabhalta for the treatment of adults with paroxysmal nocturnal hemoglobinuria. The company's CEO, Vas Narasimhan, has expressed optimism about the drug's future uptake in IgAN and C3 glomerulopathy, another progressive kidney disease.

Novartis' commitment to treating IgAN is further evidenced by its acquisition of Chinook Therapeutics for $3.2 billion, which brought two additional drug candidates, atrasentan and zigakibart, into its pipeline, both of which are currently in Phase III trials. The development and potential approval of Fabhalta, along with these other candidates, underscore Novartis' dedication to advancing treatments for kidney diseases and improving patient outcomes.